Rouan F, Pulkkinen L, Jonkman M F, Bauer J W, Cserhalmi-Friedman P B, Christiano A M, Uitto J
Department of Dermatology and Cutaneous Biology, Jefferson Medical College, and Jefferson Institute of Molecular Medicine, Thomas Jefferson University, Philadelphia, Pennsylvania 19107, USA.
J Invest Dermatol. 1998 Dec;111(6):1210-3. doi: 10.1046/j.1523-1747.1998.00422.x.
The dystrophic forms of epidermolysis bullosa (DEB) are due to mutations in the type VII collagen gene (COL7A1). In dominant DEB, a characteristic genetic lesion is a glycine substitution mutation within the collagenous domain of the protein. In this study, we have examined the molecular basis of six new families in which the proband has clinical features and/or ultrastructural findings consistent with DEB. The results revealed a glycine substitution mutation in all six families, four of which are novel and previously unpublished. In three families with clinically unaffected parents, de novo mutations G2043R and G2040V were found. These results emphasize the predominance of glycine substitution mutations in dominant DEB, and indicate that in some cases the phenotype is due to de novo dominant mutations.
营养不良型大疱性表皮松解症(DEB)是由VII型胶原蛋白基因(COL7A1)突变引起的。在显性DEB中,一个特征性的基因损伤是该蛋白胶原结构域内的甘氨酸替代突变。在本研究中,我们检查了六个新家族的分子基础,其中先证者具有与DEB一致的临床特征和/或超微结构发现。结果显示,所有六个家族均存在甘氨酸替代突变,其中四个是新的且此前未发表过。在三个父母临床未受累的家族中,发现了新发突变G2043R和G2040V。这些结果强调了甘氨酸替代突变在显性DEB中的主导地位,并表明在某些情况下,表型是由新发显性突变所致。